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Rare Disease Drug Pipeline and Clincial Trials

We understand that every scientific advancement counts when it comes to bringing new medicines to those in need. Of the 7,000 known rare diseases, less than 5% have an approved treatment option.1 This gap in care has spurred a sense of urgency—to find new, potentially life-changing approaches today.


Pfizer Rare Disease merges pioneering science with a deep understanding of the underlying disease pathology to deliver innovative treatments. With over three decades of experience in rare disease, our broad global rare disease portfolio aims to address the unmet medical needs across a number of therapeutic areas, including hematology, neurology, endocrinology, cardiology, and inherited metabolic diseases.

Work with Us

If you’re interested in collaborating with our Rare Disease research team and want to learn more about our work, visit our Rare Diseases Partnering page. We welcome the opportunity to discuss how we can work together. 


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12. Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve. 2007;(36):411-423.

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14. NIH National Human Genome Research Institute. About Duchenne Muscular Dystrophy. Accessed February 10, 2020.

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